February 2013 Blog Posts (18)

This year marks the sixth annual Rare Disease Day. This is an international event with various organizations planning awareness-raising activities. PPMD is excited to be honoring this important day by participating in a few different panels and workshops where…

The phone rings at 6AM. Something else happens inside, a feeling that something is horribly wrong. This morning, the window of my blackberry said “BRIAN D” – Brian Denger. I imagined Brian running through the snow, nails in his sneakers to cut through the ice on the streets in Biddeford Maine.

By now you’ve probably heard about PPMD’s Risk/Benefit in Duchenne Therapies program, which aims to inform the FDA and other government agencies, biopharmaceutical companies, and others about the treatment priorities and risk tolerance of the Duchenne community. We’ve had a great response to our first survey, with more than 90 participants so…

The Muscular Dystrophy Association, Parent Project Muscular Dystrophy and the Foundation to Eradicate Duchenne have called on the U.S. Senate and the U.S. House of Representatives to reauthorize the MD CARE Act of 2001, and to continue federal support for the accelerated pace of research and treatment development for muscular dystrophy. We are urging the public to contact their U.S. Senators and ask them to vote for reauthorization of …

guess for me that finding out dezi had dmd was ok... it didnt really bother me i just saw him as my son nothing different maybe it was cuz he is the 7th boy in our family to have it so it was no surprize... i do have good days and bad days more good then bad .....with dmd and work and go through a divorce and re marry to someone that is so much help in our lives .... thanks for listening

In June 2012, PPMD sponsored the first Transforming Duchenne Care meeting. The meeting was organized in response to clinical care discrepancies that exist between centers treating patients with Duchenne/Becker muscular dystrophy in North America. The purpose of this…

The Duchenne community has entered a new era where more clinical trials are coming online and the possibility of treatment is becoming a reality. Upon completion of a trial, the FDA weighs the evidence presented. But at the end of the day, it is often a value judgment about potential benefits and risks to the patient.

PPMD has launched a program – Benefit/Risk in Duchenne Therapies – to help inform the FDA and other government agencies, biopharmaceutical…

Over the last few years, we have seen something rather phenomenal happen... Our Annual Advocacy Conference use to be made up of parents and relatives of people with Duchenne. But every year, more and more people with Duchenne are joining us on Capitol Hill to tell their own story. Tayjus Surampudi, a…

Emergencies always happen when you are away from home, after “office hours,” or over a holiday. Due to this helpful timetable, it is likely that, when an emergency occurs, patients will be seen by professionals who have no knowledge of them or of their (rare) disease. During a respiratory emergency, the first reaction of emergency personnel is to give oxygen. Giving oxygen, without proper monitoring, can be life threatening to a person with Duchenne. Several such emergencies happened this…

Other than Downton Abbey, TV is not my favorite sport. On occasion, I have watched CSI, The Closer, Bones, but, truth be told, they are all the same. Person murdered, investigation, fingers point one way or another, murderer confesses. End of story.

When my boys were diagnosed with Duchenne, I fell into the same trap. Who is responsible? Both my parents smoked, so it’s their fault? My father’s company made doctor bags (Schell Leather Goods). As a child, I would ride to the…

Thanks to everyone who contributed so generously to PPMD’s holiday appeal we have been able to fund three new projectsdesigned to speed the progress of the clinical trials underway in Duchenne. As many of…

In case you didn’t know, wheelchair vans aren’t built as wheelchair vans. Chrysler, Dodge, Ford, and Volkswagen do not have wheelchair van departments that design and construct handicap accessible vans. So how are you able to purchase a wheelchair van?

Parent Project Muscular Dystrophy (PPMD) announced that it has funded $59,000 to Krista Vandenborne, PhD of the University of Florida in Gainesville to test the ability of magnetic resonance imaging (MRI) to detect changes in the muscles of those living with …

Leiden, The Netherlands – 29 January 2013 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, announced it has achieved orphan drug designation in the EU and the US for all of its compounds for the treatment of Duchenne muscular dystrophy (DMD).

The European Medicines Agency (EMA) assigned orphan drug status to Prosensa’s two preclinical compounds PRO052 and PRO055 and the US Food and…